This CME/CE activity, hosted by Ozlem Goker-Alpan, MD, Co-founder and President
Lysosomal & Rare Disorders Research & Treatment Center (LDRTC) and Neal J Weinreb, MD, FACP, Voluntary Associate Professor of Human Genetics, University of Miami Miller School of Medicine, highlights how enzyme replacement therapies (ERTs) and substrate reduction therapies (SRTs) for lysosomal storage disorders have transformed, and will continue to transform, the treatment landscape for these rare conditions.

At the end of this activity, participants should be able to:
• Describe how ERTs/SRTs have transformed the LSD population
• Describe the new research underway to improve safety and efficacy of ERTs/SRTs
• Describe how ERTs/SRTs are addressing the problem of the blood brain barrier

Educational Support for this activity was provided by Takeda, Sanofi, and Cheisi.

To obtain CME credit, please visit https://checkrare.com/learning-center/courses/